Pursuit of a Cure - Get the Facts

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Learn more about what gene therapy is and its potential in the search for a cure for FOP.

Gene therapy is an approach to treating diseases that seeks to modify or introduce genes into a patient's body. Genes are the “blueprint” for how your body functions. People with FOP have a difference in their genes that causes excess bone to grow. Gene therapy may be a way to correct that difference.

In 2020, your support allowed the IFOPA to fund the first-ever gene therapy research grant in FOP. The grant was awarded to experienced gene therapy researchers at the University of Massachusetts. In one short year, researchers at the University of Massachusetts:

  • Identified the AAV vector that can target the right cells and tissues in FOP.
  • Introduced 3 therapeutic genes into this AAV vector testing gene addition, silencing and replacement. Among them, the AAV vector for gene replacement showed good therapeutic effects in human FOP cells.
  • Then they began to test whether this vector can reduce extra bone formation in adult FOP mice when systemically or locally delivered.
  • A consortium of gene therapy experts and FOP specialists are collaborating to help guide this FOP gene therapy research. Meet the team.
There are 2 easy ways for you to learn more about gene therapy:
  1. Watch the webinar featuring the IFOPA’s new Research Director Danielle Kerkovich, PhD to learn more about gene therapy and how it might be used for FOP.
  2. The American Society of Gene & Cell Therapy has created some wonderful tools to educate patients and families. Watch one or all of these videos from their Gene & Cell Therapy 101 educational series. The 2nd video, Viral Vectors Overview, will talk about AAV Vectors which are being studied at UMass to better understand their use in FOP.

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